Deep Understanding Of Cell And Gene Therapy Genome Editing Protocols Enabled With Single-Cell Sequencing

Cell and gene therapy is revolutionizing the treatment of many intractable genetic disorders, including hemophilia, sickle-cell anemia, Huntington’s disease, and cancer. With these therapies, patients get a reprieve from life-threatening diseases or finally find relief from life-long, debilitating symptoms. Cell and gene therapy repairs genetic errors in a population of cells. After the cells are edited, they are placed in the patient where they either begin replacing the dysfunctional cells or provide an immune response against cancerous cells.

Before genetically modified cells can be placed in a patient, they must undergo rigorous testing to identify both desirable and undesirable edits. For the genome editing approach, careful selection and optimization of genome editing protocols, including the selection and design of guide RNAs (gRNAs), is an important initial step when creating cell and gene therapies. It is the selection of the right gRNAs and the characterization of the overall genome editing protocol that is critical for getting cell and gene therapies to market faster.

Single-cell sequencing technology offers exciting new capabilities for the development of cell and gene therapies. By simultaneous assessing desired and undesired editing events at the single-cell level, researchers can better optimize their protocols to build both in vivo and ex vivo cell and gene therapies.